Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies

Stavrou, Marina; Sargiannidou, Irene; Georgiou, Elena; Kagiava, Alexia; Kleopa, Kleopas A.

doi:10.3390/ijms22116048

Open AccessReview

Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies

by

Marina Stavrou

¹

,

Irene Sargiannidou

¹,

Elena Georgiou

¹,

Alexia Kagiava

¹

and

Kleopas A. Kleopa

^1,2,*

¹

Neuroscience Department, The Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, Nicosia 2371, Cyprus

²

Center for Neuromuscular Diseases, The Cyprus Institute of Neurology and Genetics, Cyprus School of Molecular Medicine, Nicosia 2371, Cyprus

^*

Author to whom correspondence should be addressed.

Int. J. Mol. Sci. 2021, 22(11), 6048; https://doi.org/10.3390/ijms22116048

Submission received: 28 April 2021 / Revised: 29 May 2021 / Accepted: 31 May 2021 / Published: 3 June 2021

(This article belongs to the Special Issue New Pharmacological Approaches for Rare Diseases)

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Abstract

Inherited neuropathies known as Charcot-Marie-Tooth (CMT) disease are genetically heterogeneous disorders affecting the peripheral nerves, causing significant and slowly progressive disability over the lifespan. The discovery of their diverse molecular genetic mechanisms over the past three decades has provided the basis for developing a wide range of therapeutics, leading to an exciting era of finding treatments for this, until now, incurable group of diseases. Many treatment approaches, including gene silencing and gene replacement therapies, as well as small molecule treatments are currently in preclinical testing while several have also reached clinical trial stage. Some of the treatment approaches are disease-specific targeted to the unique disease mechanism of each CMT form, while other therapeutics target common pathways shared by several or all CMT types. As promising treatments reach the stage of clinical translation, optimal outcome measures, novel biomarkers and appropriate trial designs are crucial in order to facilitate successful testing and validation of novel treatments for CMT patients.

Keywords: Charcot-Marie-Tooth disease; inherited neuropathy; gene therapy; axonal degeneration; biomarkers

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MDPI and ACS Style

Stavrou, M.; Sargiannidou, I.; Georgiou, E.; Kagiava, A.; Kleopa, K.A. Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies. Int. J. Mol. Sci. 2021, 22, 6048. https://doi.org/10.3390/ijms22116048

AMA Style

Stavrou M, Sargiannidou I, Georgiou E, Kagiava A, Kleopa KA. Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies. International Journal of Molecular Sciences. 2021; 22(11):6048. https://doi.org/10.3390/ijms22116048

Chicago/Turabian Style

Stavrou, Marina, Irene Sargiannidou, Elena Georgiou, Alexia Kagiava, and Kleopas A. Kleopa. 2021. "Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies" International Journal of Molecular Sciences 22, no. 11: 6048. https://doi.org/10.3390/ijms22116048

APA Style

Stavrou, M., Sargiannidou, I., Georgiou, E., Kagiava, A., & Kleopa, K. A. (2021). Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies. International Journal of Molecular Sciences, 22(11), 6048. https://doi.org/10.3390/ijms22116048

Note that from the first issue of 2016, this journal uses article numbers instead of page numbers. See further details here.

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Emerging Therapies for Charcot-Marie-Tooth Inherited Neuropathies

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